Chordoma Foundation

Comprehensive drug screening effort offers the best chance of finding new treatments for chordoma in the near-term

We’re excited to announce that the Chordoma Foundation and the Broad Institute of Harvard and MIT recently embarked upon the most thorough effort yet to identify existing drugs with the potential to provide new treatments for chordoma patients.

Through this year-long project a team of Broad Institute researchers led by Drs. Stuart Schreiber and Tanaz Sharifnia will screen chordoma cells with the Drug Repurposing Hub collection of approximately 6,000 existing drugs – the world’s most complete set of drugs that have been FDA approved or have entered clinical trials. Any drugs demonstrating compelling activity against chordoma cells have the potential to rapidly advance into the clinic, offering the quickest path to finding new treatments for patients in need of better options.

Supported by a $300,000 grant from the Foundation, this drug repurposing screen builds upon five years of Foundation-funded target discovery work in the Schreiber lab at the Broad Institute. This previous work culminated last year in the publication of decisive evidence that the protein brachyury is, in fact, chordoma’s Achilles’ heel, and revealed a new way to turn brachyury off and block chordoma cell growth.

The Schreiber lab’s prior experience in screening 500 drugs and other chemicals for activity in chordoma cell lines will place them in an excellent position to be able to complete screening of the full library of 6,000 drugs later this year. In parallel with the drug repurposing screen, the Schreiber team is also mining the vast dataset they have generated using complementary genetic approaches to identify additional chordoma dependencies and new potential drug targets.

The promise of repurposing drugs

Drug repurposing is a drug development strategy predicated on the reuse of existing drugs for new medical indications. While the process of bringing new cancer treatments from bench to bedside can be slow and expensive, taking an existing drug and seeing whether it can be used as an effective treatment for another condition saves time, cost, and in many cases, lives.

Earlier this year, for example, a major study conducted through the Broad’s Drug Repurposing Hub found that nearly 50 non-oncology drugs which were either approved or in clinical trials for a variety of other conditions – ranging from diabetes to inflammation to arthritis – also had the ability to kill cancer cells. This study marked the first time that researchers screened the Broad’s large collection of mainly non-cancer drugs for their anti-cancer capabilities and underscores the promise it offers chordoma and other rare cancers to identify new treatment options in the near-term.  This promise is why drug repurposing is one of our top five strategic priorities in 2020.

Rapid translation to the clinic

If successful, by the end of 2020, this screening effort will identify multiple drugs that will proceed for additional testing in chordoma mouse models through our Drug Screening Program. If activity is observed in the mouse models, the drugs can then rapidly progress to clinical testing in humans.

We are very excited to support this program and look forward to keeping you updated on its progress throughout the year.

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